The effects of intracisternal enzyme replacement versus sham treatment on central neuropathology in preclinical canine fucosidosis
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- 作者：Gauthami Sudhamayee Kondagari ; Jessica Louise Fletcher…
- 关键词：Fucosidosis ; Intracisternal enzyme replacement therapy ; Neuroinflammatory markers ; Canine model ; Neurodegeneration ; CNS
- 刊名：Orphanet Journal of Rare Diseases
- 出版年：2015
- 出版时间：December 2015
- 年：2015
- 卷：10
- 期：1
- 全文大小：2,934 KB
- 刊物主题：Medicine/Public Health, general; Pharmacology/Toxicology; Medicinal Chemistry;
- 出版者：BioMed Central
- ISSN：1750-1172
文摘
Background Fucosidosis results from lack of α-L-fucosidase activity, with accumulation of fucose-linked substrates in the nervous system and viscera leading to progressive motor and mental deterioration, and death. The naturally occurring dog model of fucosidosis was used to evaluate the neuropathological responses to partial enzyme replacement, and substrate reduction in early disease following treatment with recombinant canine α-L-fucosidase delivered through cerebrospinal fluid.